About 1 in 500 infants in the U.S. is born with hearing loss, most often caused by genetic factors. Hearing aids or cochlear implants can help children hear better, but kids fitted with these devices still often have a hard time hearing sounds that are far away or in noisy environments.
Jonathon Whitton, a clinician-scientist, is leading Regeneron’s efforts to develop gene therapies to treat hearing loss in children. In October, the company announced promising results from a small trial that tested a gene therapy in children with profound hearing loss caused by a genetic deficiency of the protein otoferlin, which affects an estimated 50 newborns in the U.S. every year.
Of the 12 children who received the gene therapy, 11 experienced meaningful improvements to their hearing, including some who developed normal hearing within a few months. Whitton says it’s been tremendous to see how transformational the therapy can be. “I live elated,” he says.